Signals Blog


Welcome to your Update from the Clinic for the month of December. bluebird bio’s first four patients are transfusion-free after treatment with its gene therapy Lentiglobin. Fate Therapeutics’ hematopoietic stem cell-modulating technology ProHema seems to be on track to produce results in the clinic.

bluebird bio (BLUE), which recently priced a common offering to raise over US$200M, is having success with its gene therapy platform in patients with β-thalassemia. The company announced data for eight patients, five of whom are in the ongoing Phase 1/2 Northstar study investigating Lentiglobin BB305 for β-thalassemia, while the other three are from a study investigating the same gene therapy product for sickle cell disease.

After three months of follow-up, four patients with β-thalassemia have been declared transfusion-free following treatment. BB305 is an autologous product composed of a patient’s own CD34+ bone marrow stem cells transduced with a vector carrying the gene for human beta globin (a protein that makes up the most common form of hemoglobin in humans). See the data in this press release.

Early mortality following bone marrow transplant can result if hematopoietic stem cells fail to reconstitute the immune system in a timely manner; in such a scenario patients become highly vulnerable to opportunistic pathogens, including bacteria, yeast, and fungus. This is particularly the case when stem cells are derived from cord blood, which are more primitive than those found in the bone marrow or peripheral blood, and require more time to repopulate the bone marrow compartment.

Fate Therapeutics (FATE), which is mid-way through a Phase 2 study (PUMA) investigating ProHema, an ex vivo expanded hematopoietic stem cell product modulated with small molecules to improve proliferative ability and survival, released positive data showing that its modulation technology can reduce the time to engraftment by six days relative to historical data. Patients in the study are either receiving two un-manipulated cords, or one ProHema-modulated cord and one un-manipulated cord.

Athersys (ATHX) continues to push forward with MultiStem and has completed enrolment in its Phase 2 study investigating the product for ischemic stroke. Initial results from the study should be available at the end of the first quarter of 2015.

StemCells Inc. (STEM) treated its first patient with its Hu-CNS-SC cell therapy product for cervical spinal cord injury (Pathway study). This is the first study to investigate the efficacy of human neural stem cells for treating spinal cord injury.

Stemline Therapeutics (STML), one of three up-and-coming cancer stem cell companies, completed enrolment of a Phase 1/2 study investigating its antibody SL-401 in patients with one of four rare myeloproliferative neoplasms. SL-401 targets the interleukin-3 receptor (IL-3R).

It will be interesting to see how FATE’s ex vivo modulation paradigm for cord blood stem cells compares to the ex vivo expansion technologies currently being developed by companies such as Novartis and Gamida Cell (see an overview in this blog). While expansion aims to reduce the time to reconstitution of the blood and immune systems by increasing the number of cells that are transplanted, ProHema works by creating a form of “super” stem cell that divides more rapidly in vivo and has improved survival abilities.

Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit







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Mark Curtis

Mark Curtis

Mark is a Business Development Analyst at the Centre for Commercialization of Regenerative Medicine (CCRM), where he collaborates with the team to help evaluate the commercial potential of regenerative medicine and cell therapy technologies. He began his career at Princess Margaret Hospital studying cellular reprogramming of human skin cells. Following this project, he left the laboratory and took on a role with Bloom Burton & Co., a healthcare-focused investment dealer. While there he supported the advisory team in carrying out scientific diligence on early-stage biotechnology companies. Prior to joining CCRM he was a consultant to Stem Cell Therapeutics, a Toronto-based biotechnology company focused on developing therapeutics targeting cancer stem cells. Mark received a Master’s degree from the University of New South Wales in Sydney, where he studied the directed differentiation of embryonic stem cells, and a Bachelor’s degree in Biology, from Queen’s University. Follow Mark on Twitter @markallencurtis
Mark Curtis

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